Neuroblastoma Research & Treatment Innovations

The Neuroblastoma Advanced Therapies Center at Cincinnati Children’s is a leading center for the development and clinical testing of new approaches to neuroblastoma. Our physicians and laboratory scientists work together to speed research breakthroughs from the laboratory to the clinic setting. Current clinical research initiatives in neuroblastoma focus on the development of new treatments using:

Novel targeted therapies

New approaches to high-dose chemotherapy

Targeted radiopharmaceutical therapies

Our Cancer Program also has a robust clinical research effort to treat children with chemotherapy-resistant neuroblastoma using targeted radiation therapies. For example, Cincinnati Children’s is one of just five cancer centers testing 131I-MIBG therapy on pediatric patients with relapsed neuroblastoma. This therapy involves injecting a chemical (MIBG) into the bloodstream in combination with radioactive iodine. The chemical is absorbed by neuroblastoma cells, thereby facilitating killing of the tumor cells by the radioactive iodine.

Very few centers have the infrastructure to conduct intensive, technically demanding trials of this nature. Brian D. Weiss, MD, leads this clinical research effort at Cincinnati Children’s, and is also leading national efforts through his involvement with Children’s Oncology Group.

Cincinnati Children’s researchers are also investigating novel agents or combinations of agents to treat the most difficult forms of relapsed or refractory neuroblastoma. A number of Cincinnati Children’s pilot projects have subsequently been developed into national trials by the Children’s Oncology Group and other national clinical research consortia. An example of this type of research includes the work of Lars M. Wagner, MD, who is recognized as an expert in the development of regimens incorporating the important drugs temozolomide and irinotecan, as well as on potential approaches to overcoming potential resistance to these combinations.

National Referral Center

Cincinnati Children’s is a major referral center for the National Cancer Institute’s two new pediatric research initiatives for children with high-risk neuroblastoma: the Pediatric Phase I Consortium and the New Approaches to Neuroblastoma Therapy Consortium. Many of our physicians serve in leadership positions for these and other pediatric cancer organizations, further strengthening our link to the latest clinical research opportunities.